Search | Clinical Trials Finder

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Nov 14, 2026, 9:47 PM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 413 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Rare Disease Clear all

Conditions: Alagille Syndrome, Cholestasis, Biliary Atresia
Interventions: magnesium gluconate, magnesium sulfate; Drug
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 3 Years to 18 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2000
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Ewing Sarcoma, Ewing Family of Tumors, Ewing's Tumor Metastatic, Ewing's Sarcoma Metastatic, Ewing's Tumor Recurrent, Rare Diseases, Sarcoma, Neoplasms, Connective and Soft Tissue, Neoplasms by Histologic Type, Neoplasms, Bone Tissue, Neoplasms, Connective Tissue, Sarcoma, Ewing, Neoplasms
Interventions: Vigil, Irinotecan, Temozolomide; Biological · Drug
Lead sponsor: Gradalis, Inc.; Industry
Eligibility: 2 Years and older
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 15
States / cities: Little Rock, Arkansas • Los Angeles, California • Jacksonville, Florida + 12 more

Conditions: Choroideremia
Interventions: No Intervention; Other
Lead sponsor: Biogen; Industry
Eligibility: 10 Years and older
Enrollment: 1,178 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Non Small Cell Lung Cancer
Interventions: osimertinib; Drug
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 3
States / cities: Durham, North Carolina • Columbus, Ohio • Pittsburgh, Pennsylvania

Conditions: Cystinosis
Interventions: Cystagon® (Cysteamine Bitartrate), Cysteamine Bitartrate Delayed-release Capsules (RP103); Drug
Lead sponsor: Amgen; Industry
Eligibility: 6 Years and older
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2011
U.S. locations: 3
States / cities: Stanford, California • Atlanta, Georgia • Chicago, Illinois

Conditions: Undiagnosed Diseases, Cardiovascular Disease
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 1 Month to 100 Years
Enrollment: 10,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2050
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Genetic Disease
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years and older
Enrollment: 157 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2014
U.S. locations: 2
States / cities: Bethesda, Maryland • Strasburg, Pennsylvania

Conditions: Primary Ciliary Dyskinesia
Interventions: Not listed
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 0 Years and older
Enrollment: 180 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2000
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions: Parent-Infant Inter(X)action Intervention (PIXI); Behavioral
Lead sponsor: RTI International; Other
Eligibility: Up to 99 Years
Enrollment: 120 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

Conditions: Homozygous Familial Hypercholesterolemia (HoFH)
Interventions: Not listed
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 1
States / cities: Boca Raton, Florida

Conditions: Rare Diseases
Interventions: Not listed
Lead sponsor: University of Kentucky; Other
Eligibility: 1 Minute to 101 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 1
States / cities: Lexington, Kentucky

Conditions: Cystinosis
Interventions: RP103; Drug
Lead sponsor: Amgen; Industry
Eligibility: Up to 6 Years
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Cystic Fibrosis
Interventions: glucose, insulin, leucine; Drug
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 0 Years and older · Female only
Enrollment: 36 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 2001
U.S. locations: 1
States / cities: Salt Lake City, Utah

Conditions: Neuroblastoma, Medulloblastoma, Brain Tumors, Rare Tumors
Interventions: Guided Therapy- Pediatric Gene Analysis Platform; Device
Lead sponsor: Giselle Sholler; Other
Eligibility: 13 Months and older
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2024
U.S. locations: 13
States / cities: Phoenix, Arizona • San Diego, California • Hartford, Connecticut + 10 more

Conditions: Microvillus Inclusion Disease, Congenital Disorders, Rare Diseases
Interventions: Crofelemer Powder for Oral Solution, Placebo Powder for Oral Solution; Drug
Lead sponsor: Napo Pharmaceuticals, Inc.; Industry
Eligibility: Up to 17 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Friedreich's Ataxia (FA)
Interventions: SGT-212; Drug
Lead sponsor: Solid Biosciences Inc.; Industry
Eligibility: 18 Years to 40 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 3
States / cities: Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania

Conditions: Cancer
Interventions: Dabrafenib, Trametinib; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 206 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2021
U.S. locations: 8
States / cities: Little Rock, Arkansas • Santa Monica, California • Bethesda, Maryland + 4 more

Conditions: Fabry Disease
Interventions: ST-920; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2025
U.S. locations: 9
States / cities: Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more

Conditions: Osteoporosis
Interventions: Alendronate; Drug
Lead sponsor: Medical University of South Carolina; Other
Eligibility: 5 Years to 14 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2008
U.S. locations: 1
States / cities: Charleston, South Carolina

Conditions: Multiple Myeloma
Interventions: Not listed
Lead sponsor: Icahn School of Medicine at Mount Sinai; Other
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 3
States / cities: San Francisco, California • New York, New York

Conditions: Cushing's Syndrome
Interventions: Not listed
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 20 Years to 60 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1994
U.S. locations: 1
States / cities: Ann Arbor, Michigan

Conditions: Retinal Artery Occlusions, Polypoidal Choroidal Vasculopathy, Retinal Arterial Macroaneurysm, Juxtafoveal Telangiecasia, Central Serous Chorioretinopathy
Interventions: Not listed
Lead sponsor: Oregon Health and Science University; Other
Eligibility: 7 Years to 100 Years
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2026
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Healthy
Interventions: Dichloroacetate (DCA), GSTZ1 haplotyping; Drug · Other
Lead sponsor: University of Florida; Other
Eligibility: 18 Years to 70 Years
Enrollment: 45 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2017
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Non-Small Cell Lung Cancer, Advanced Non-Small Cell Squamous Lung Cancer, Metastatic Lung Non-Small Cell Carcinoma, Metastatic Lung Cancer, NSCLC, Advanced Lung Carcinoma, Epidermal Growth Factor Receptor C797S, Epidermal Growth Factor Receptor G719X, EGF-R Positive Non-Small Cell Lung Cancer, EGFR-TKI Resistant Mutation
Interventions: silevertinib (BDTX-1535) monotherapy; Drug
Lead sponsor: Black Diamond Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 25
States / cities: Birmingham, Alabama • Gilbert, Arizona • Huntington Beach, California + 20 more

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Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease

A Trial For Participants With Ewing's Sarcoma Treated With Vigil in Combination With Irinotecan and Temozolomide

Natural Disease Progression in Participants With Choroideremia

A Study Osimertinib in Patients With Stage 4 Non-small Cell Lung Cancer With Uncommon EGFR Mutations

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

Human Biospecimen Procurement Protocol: Biorepository to Support Translational Research to Identify Disease Mechanism(s)

Genetic Studies in the Amish and Mennonites

Genetic Study of Patients With Primary Ciliary Dyskinesia

Parent and Infant Inter(X)Action Intervention (PIXI)

Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

Finding Genes for Rare Diseases

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

Study of Metabolic Effects of Pregnancy in Women With Cystic Fibrosis

Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer

Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

Efficacy and Safety of the Combination Therapy of Dabrafenib and Trametinib in Subjects With BRAF V600E- Mutated Rare Cancers

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

Phase II Study of Alendronate Sodium in Children With High-Turnover Idiopathic Juvenile Osteoporosis

Delayed Toxicities Post-CAR-T

Study of Depression, Peptides, and Steroids in Cushing's Syndrome

OCT in Rare Chorioretinal Diseases

A Pilot Study of Rapid Haplotyping Procedure for Personalized Dosing of Dichloroacetate (DCA) in Healthy Volunteers

Phase 1/2 Study of Silevertinib (BDTX-1535) in Patients With Glioblastoma or Non-Small Cell Lung Cancer With EGFR Mutations